Gladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA. Department of Pharmaceutical Chemistry, University of California-San Francisco, San Francisco, CA 94158, USA.
Gladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Science. 2016 Jun 3;352(6290):1216-20. doi: 10.1126/science.aaf1502. Epub 2016 Apr 28.
Reprogramming somatic fibroblasts into alternative lineages would provide a promising source of cells for regenerative therapy. However, transdifferentiating human cells into specific homogeneous, functional cell types is challenging. Here we show that cardiomyocyte-like cells can be generated by treating human fibroblasts with a combination of nine compounds that we term 9C. The chemically induced cardiomyocyte-like cells uniformly contracted and resembled human cardiomyocytes in their transcriptome, epigenetic, and electrophysiological properties. 9C treatment of human fibroblasts resulted in a more open-chromatin conformation at key heart developmental genes, enabling their promoters and enhancers to bind effectors of major cardiogenic signals. When transplanted into infarcted mouse hearts, 9C-treated fibroblasts were efficiently converted to chemically induced cardiomyocyte-like cells. This pharmacological approach to lineage-specific reprogramming may have many important therapeutic implications after further optimization to generate mature cardiac cells.
重编程体成纤维细胞为其他谱系将为再生治疗提供有前途的细胞来源。然而,将人类细胞转分化为特定的同质、功能性细胞类型具有挑战性。在这里,我们表明通过用我们称之为 9C 的九种化合物组合处理人成纤维细胞,可以产生类心肌细胞。化学诱导的类心肌细胞均匀收缩,在转录组、表观遗传和电生理特性上与人心肌细胞相似。9C 处理人成纤维细胞导致关键心脏发育基因的染色质构象更加开放,使它们的启动子和增强子能够结合主要心脏生成信号的效应物。当移植到梗死的小鼠心脏中时,9C 处理的成纤维细胞被有效地转化为化学诱导的类心肌细胞。这种针对谱系特异性重编程的药物治疗方法在进一步优化以产生成熟的心脏细胞后,可能具有许多重要的治疗意义。